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If it's good enough for mice, it's good enough for me...

Hi there,

I'm new to this forum (and to the whole dying thing), so forgive me if I make any faux pas...

I was diagnosed with MND/ALS in June. At the moment it's my left hand and arm that are affected (muscle wastage, weak hand and fingers), along with the standard twitching there and elsewhere (although I like to think of it as having rippling muscles)...

Following a period of resignation to my fate - (it's incurable, right?) - having read a few of the threads on here, I have decided to participate in my own clinical trial(s)...

There are a couple of drugs that I have read have had excellent results with mice, but have not yet been approved for testing on humans, despite the fact that they are FDA approved for use with other illnesses. (USDA (ursodeoxycholic acid) and Peramanel, currently used for treating kidney disease and epilepsy respectively).

I particularly liked this bit... 'Researchers at the University of Tokyo demonstrated that the FDA-approved anti-epileptic drug perampanel is able to prevent the progression of amyotrophic lateral sclerosis (ALS) and normalize the death of motor neurons in an animal model of the disease. The findings indicate the drug may be a potential candidate for ALS treatment and should be explored in clinical trials.'

Well that's promising, but time isn't on my side, so I'm heading to the world of online pharmacies and am going to start testing. I realise that a trial of one subject will mean that any reduction in the speed of my decline will be virtually impossible to verify, but any improvements may well be measurable.

So, I'm asking the following questions...

1. Anyone up for joining me? I figure we don't have much to lose at this point.
2. Anyone have any insights? (Amounts to take, frequency, etc)
3. Anyone have any ideas of how I could scientifically measure any progress
4. Anyone know of any reputable outlets for these drugs (I realise that the internet isn't always the most reliable source of medication...)

Good luck, and the very best to you in your clinical trial. I am curious to know though how do you go about getting your own drugs ?? Are these drugs over the counter medications or do you need a doctor's prescription ? If they are illegal drugs be careful what your taking side effects could be potentially dangerous.

You'll have to see if your GP will prescribe it "off label". Perampanel is subject to additional monitoring, so you may have difficulty getting your hands on it. You'll need to decide on the dose of Perampanel yourselves as known doses are effective in treating epilepsy only.

It's difficult to quantify whether any treatment is slowing progression, as it isn't linear, so nobody really knows for sure how they would have progressed anyway!

I'm new to this forum (and to the whole dying thing), so forgive me if I make any faux pas...

I was diagnosed with MND/ALS in June. At the moment it's my left hand and arm that are affected (muscle wastage, weak hand and fingers), along with the standard twitching there and elsewhere (although I like to think of it as having rippling muscles)...

Following a period of resignation to my fate - (it's incurable, right?) - having read a few of the threads on here, I have decided to participate in my own clinical trial(s)...

There are a couple of drugs that I have read have had excellent results with mice, but have not yet been approved for testing on humans, despite the fact that they are FDA approved for use with other illnesses. (USDA (ursodeoxycholic acid) and Peramanel, currently used for treating kidney disease and epilepsy respectively).

I particularly liked this bit... 'Researchers at the University of Tokyo demonstrated that the FDA-approved anti-epileptic drug perampanel is able to prevent the progression of amyotrophic lateral sclerosis (ALS) and normalize the death of motor neurons in an animal model of the disease. The findings indicate the drug may be a potential candidate for ALS treatment and should be explored in clinical trials.'

Well that's promising, but time isn't on my side, so I'm heading to the world of online pharmacies and am going to start testing. I realise that a trial of one subject will mean that any reduction in the speed of my decline will be virtually impossible to verify, but any improvements may well be measurable.

So, I'm asking the following questions...

1. Anyone up for joining me? I figure we don't have much to lose at this point.
2. Anyone have any insights? (Amounts to take, frequency, etc)
3. Anyone have any ideas of how I could scientifically measure any progress
4. Anyone know of any reputable outlets for these drugs (I realise that the internet isn't always the most reliable source of medication...)

I'd be grateful for any comments, insights or advice!

Kind regards,

Kuiper

Kuiper:
I am up for trying it. Please let us know where to source the muti when you do.
This is an instance where I have a clear answer to the question "Are you a mouse or a man?"

1) You are right, we don't have much to lose and many of us would try anything with some promise.

2) You can find the recommend amounts etc from a drug book that the doctor and pharmacy use, and probably on a NHS web site. I believe that I still have an older one of these books.

3) To scientifically measure any progress is extremely hard to do with ALS. Riluzole and a placibo were given to a group of people and it showed some improvement but without doing very large group there accuracy can be very poor. Everyones rate of progression is different and sometime it appears to stop for a while. If you do try something and it appears to stop progression then that would be very positive. You can monitor the areas that are affected by logging certain things down every week.

4) You could ask your Doctor and explain the reasons why. Also you could ask your specialist. I don't think that we can buy directly but if you know anyone taking them??? Maybe different countries have different rules.

Best wishes, Terry

Last edited by Terry; 13th July 2016 at 00:38.

TB once said that "The forum is still the best source for friendship and information."

It will only remain so if new people post and keep us updated on things that work or don't work and tips.

Please post on old threads that are of use so that others see them and feel free to start new subjects and threads.

ccinjersey - They are both medicinal drugs already in use for other ailments (as opposed to illegal), but not over the counter either. They'll either need to be sourced on the internet or 'off-label', which I didn't even know was a thing...

Ellie - can you clarify the off-label thing? How would this work? Can I ask my GP for any drug I like, essentially?

With doses, I guess there's no point in taking half-pills... I'll take the highest, safest dose I suppose...

It's a real pain in the bum that we all have such differing rates of progression... It's no wonder all my questions to the consultant et al are met with the same response - 'We don't know'...

Terry - Thanks for the pointers - You posted while I was typing I had originally thought that I'd be buying from abroad, but I'll be asking my GP and Consultant first, now! I don't know anyone already on them, all my friends are sickeningly healthy!

Kuiper. Welcome to the forum. I am on my left side like you and was formally diagnosed May 2014. You can look up a lot things on this forum and get a lot of information from the MND research blog. There is research world wide and two years ago read article in the paper and Professor Linda Greensmith of UCL was working on mice and the article stated that she hoped the first test operations in Mnd patients could start within five years.
I will be very happy when they run out of mice and want to start on me. As for drugs I take riluzole like most of us but would not go buying anything else that could be very dangerous.
Jay Tee

A fellow lefty, eh? I see from your join date that you've been here for a couple of years - Can I ask, is your progress quite slow, or is mine quite fast? (comparatively speaking)... I only had a twitchy finger in Feb and now my hand is quite skeletal and is really weak, typing is a struggle etc.

Anyway, I always liked the poem 'Do not go gentle into that good night' by Dylan Thomas, and I had thought that I'd been robbed of my opportunity to fight death, tooth and nail.... I don't want to take my last breath thinking I didn't at least try...

Bring on the cure, bring on the operations, bring on the wine and the women... Okay, I'll settle for the cure and the operations...

Hi Kuiper,
Welcome and sorry you find yourself here. Glad you are not going to take this quietly and good luck with your experimentation. If you take a look at the alstdi forum which is USA based with members like yourself seeking a solution. Mnd is referred to as ALS in America. They can probably give you advice on dosages as well. There is a move to give people suffering from illnesses without a known cure a right to try in the USA, maybe time to push for this in the uk.